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Cost-Effectiveness of Transfusions to Prevent Silent Cerebral Infarcts in Children with Sickle Cell Disease

December 29, 2021

Guidelines published by the American Society of Hematology in 2020 recommend annual transcranial doppler ultrasound (TCD) screening to help identify silent cerebral infarcts in children with severe forms of sickle cell disease (SCD).  In children with abnormal TCD screenings, the guidelines recommend transfusions every 3-4 weeks for at least a year to reduce the risk of further silent strokes.  These silent but debilitating strokes occur in almost 40% of children with severe SCD by 18 years of age and can reduce the quality of life.  To better understand the effectiveness of transfusions for these children, in terms of costs and prevention of recurrent cerebral infarcts, researchers followed 196 children with severe SCD for three years (mean age 10.0 years, 43% male). During the study, 196 children were randomly assigned to receive either monthly transfusions (N=90) or standard care (N=106).  Six patients in the transfusion arm had a cerebral infarct vs 16 in the standard care arm; patients in the transfusion arm had fewer hospitalizations and shorter stays.  The average direct medical costs and indirect educational costs were $282,427 and $62,178 for the transfusion and standard care groups, respectively.  The cost of transfusion and chelation therapy greatly exceeded savings in hospitalizations, but quality of life was not measured.  Further research is needed on how to cost-effectively improve the quality of life for these patients.

Reference:

Hsu P, Gay JC, Lin CJ, Rodeghier M, DeBaun MR, and RM Cronin.  Economic evaluation of regular transfusions for cerebral infarct recurrence in the silent cerebral infarct transfusion trial.  Blood Advances 2021; 5(23); 5032-5040

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